CA2971920C - Rnai induced huntingtin gene suppression - Google Patents

Rnai induced huntingtin gene suppression Download PDF

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Publication number
CA2971920C
CA2971920C CA2971920A CA2971920A CA2971920C CA 2971920 C CA2971920 C CA 2971920C CA 2971920 A CA2971920 A CA 2971920A CA 2971920 A CA2971920 A CA 2971920A CA 2971920 C CA2971920 C CA 2971920C
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sequence
rna
seq
double stranded
mirna
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French (fr)
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CA2971920A1 (en
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Pavlina Stefanova KONSTANTINOVA
Jana Miniarikova
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Uniqure IP BV
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA2971920A 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression Active CA2971920C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14200308.6 2014-12-24
EP14200308 2014-12-24
PCT/EP2015/081157 WO2016102664A1 (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression

Publications (2)

Publication Number Publication Date
CA2971920A1 CA2971920A1 (en) 2016-06-30
CA2971920C true CA2971920C (en) 2024-05-07

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Family Applications (1)

Application Number Title Priority Date Filing Date
CA2971920A Active CA2971920C (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression

Country Status (25)

Country Link
US (4) US10174321B2 (he)
EP (2) EP3540063A1 (he)
JP (2) JP6839094B2 (he)
KR (1) KR102636633B1 (he)
CN (1) CN108064292B (he)
AU (1) AU2015370903B2 (he)
BR (1) BR112017013573A2 (he)
CA (1) CA2971920C (he)
CO (1) CO2017007335A2 (he)
CY (1) CY1121733T1 (he)
DK (1) DK3237618T3 (he)
EA (1) EA037696B1 (he)
ES (1) ES2732023T3 (he)
HK (1) HK1246344B (he)
HR (1) HRP20190992T1 (he)
HU (1) HUE043842T2 (he)
IL (1) IL252990B (he)
LT (1) LT3237618T (he)
MX (1) MX2017008500A (he)
PL (1) PL3237618T3 (he)
PT (1) PT3237618T (he)
RS (1) RS58862B1 (he)
SI (1) SI3237618T1 (he)
TR (1) TR201908969T4 (he)
WO (1) WO2016102664A1 (he)

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Also Published As

Publication number Publication date
EA201791417A1 (ru) 2017-12-29
TR201908969T4 (tr) 2019-07-22
US20200339992A1 (en) 2020-10-29
EP3237618B1 (en) 2019-05-22
US11371044B2 (en) 2022-06-28
SI3237618T1 (sl) 2019-09-30
CO2017007335A2 (es) 2018-01-05
AU2015370903B2 (en) 2021-06-17
HUE043842T4 (hu) 2019-08-28
CA2971920A1 (en) 2016-06-30
NZ733296A (en) 2021-10-29
HUE043842T2 (hu) 2019-09-30
US20170355989A1 (en) 2017-12-14
MX2017008500A (es) 2018-02-01
WO2016102664A1 (en) 2016-06-30
EA037696B1 (ru) 2021-05-12
US20230119344A1 (en) 2023-04-20
US10174321B2 (en) 2019-01-08
IL252990A0 (he) 2017-08-31
HRP20190992T1 (hr) 2019-09-20
PL3237618T3 (pl) 2019-09-30
EP3540063A1 (en) 2019-09-18
RS58862B1 (sr) 2019-07-31
KR20170120572A (ko) 2017-10-31
BR112017013573A2 (pt) 2018-03-06
DK3237618T3 (da) 2019-06-24
JP2018502601A (ja) 2018-02-01
IL252990B (he) 2021-09-30
ES2732023T3 (es) 2019-11-20
CN108064292B (zh) 2021-05-04
US20190144860A1 (en) 2019-05-16
JP2021000136A (ja) 2021-01-07
AU2015370903A1 (en) 2017-07-20
JP6839094B2 (ja) 2021-03-03
LT3237618T (lt) 2019-07-10
US10767180B2 (en) 2020-09-08
CY1121733T1 (el) 2020-07-31
HK1246344B (zh) 2020-01-10
PT3237618T (pt) 2019-07-04
KR102636633B1 (ko) 2024-02-13
CN108064292A (zh) 2018-05-22
EP3237618A1 (en) 2017-11-01

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